Rare Disease

Amgen's PSCK9 shows rare disease potential

Amgen's PSCK9 shows rare disease potential

By

The Thousand Oaks, Calif. company says its cholesterol-lowering antibody hit its clinical trial endpoint among rare disease patients.

NICE wants more Soliris information

The UK watchdog wants to know what's behind the rare-disease drug's high price.

Sanofi seeks to revive Kynamro launch

Sanofi seeks to revive Kynamro launch

By

Kynamro's slow start, coupled with competition, has prompted Sanofi to add sales reps.

Rare diseases may be less so

Regulatory Focus discusses how the expanding US population may make it harder for a condition to qualify as rare.

Genzyme rare-disease drug gets priority label

The FDA granted priority review for the experimental, all-oral Gaucher's disease treatment.

FDA pushes review date for Biogen blood drug

The agency extended the review time for Biogen's Alprolix by three months.

FDA panel backs BioMarin rare-disease drug

FDA panel backs BioMarin rare-disease drug

By

The patient population is small, at around 3,000, but the expected price tag is $330,000 a year.

IMS predicts $1T global drug market by 2017

IMS predicts $1T global drug market by 2017

By

The catch is the difference between sales and revenues. Generics will continue to be a force, and small-audience drugs will become increasingly important.

Alexion recalls some Soliris batches

The US-only recall amounts to less than 1% of the company's inventory.

FDA delays Sarepta MD drug

The regulator says the drugmaker needs to do more testing.

Lilly partners with NIH for rare-disease research

By

Eli Lilly is the first Big Pharma to team up with the agency in its rare-disease pre-clinical development program.

Analyst says smart money is on drugs for certain orphan diseases

By

Price, market potential and emotional pull make this subcategory of orphan diseases one to watch, an analyst writes. Hint: it's not oncology.

Business briefs: Takeda, Sangamo, Catalyst, plus seniors and insurance

Takeda moves an Alzheimer's treatment to Phase III; Sangamo acquires Ceregene; Catalyst gets breakthrough designation for auto-immune disorder drug; study says seniors likely to opt out of health insurance

Business briefs: Bristol-Myers Squibb, Zafgen, GSK, Shire

Australia's price watchdogs are assessing the value of BMS cancer drug Yervoy; why a Phase II weight drug from Zafgen could open up the category; GSK halts a Crohn's trial; and Shire extends Santaris rare-disease collaboration

Novartis breakthough designation hat trick

By

The drug maker scored its third breakthrough designation label, this time for orphan drug bimagrumab, a possible treatment for a muscle-wasting disease.

Business briefs: Alexion, AMA, Eli Lilly

Alexion plans on moving into new HQ in 2015, AMA declares obesity is a disease, FDA looks into Zyprexa patient deaths

Fabry film spotlights a rare disease

Fabry film spotlights a rare disease

By

Genzyme is taking rare disease awareness to another level with a documentary film, dubbed "Facing Fabry Together," that follows four families grappling with the genetic disorder.

Rare disease awareness fails to ease physician, patient burden

By

A study shows FDA approvals and awareness efforts are having little impact on physician awareness or patient frustration.

Rare disease awareness more than just a day for pharmas

Rare disease awareness more than just a day for pharmas

By

Industry observances of Rare Disease Awareness Day are getting more elaborate of late—perhaps not solely for altruistic reasons, as a third of NMEs approved by FDA over the past five years were for the treatment of rare diseases.

Drug for ultrarare lipid disorder OK'd by FDA

Drug for ultrarare lipid disorder OK'd by FDA

By

Aegerion's Juxtapid is first to market for treating the ultrarare inherited cholesterol condition HoFH, or homozygous familial hypercholesterolemia.

Rare diseases worth the investment, study says

By

A recent study says that the financial incentives go beyond taxes and grants and that investing in R&D can mean entry into a space that's grown 26% from 2001 to 2010.

Company news: Burson-Marsteller, Corcept and Orexigen

PR firm Burson-Marsteller launched a new healthcare subsidiary, Orexigen's net loss widened in Q2, and Corcept closed out its first full quarter with orphan treatment Korlym.

Email Newsletters